Early access has become an increasingly prominent issue within the global biotech sector – how to enable patient access to pioneering but investigational new treatments. In 2014, US biotech company Chimerix made international headlines following a social media campaign urging the company to provide Josh Hardy, a critically ill seven year old boy, with access to a potentially life-saving drug. One year on, Mark Corbett, SVP Clinigen Global Access Programs (GAP), explores the importance of early access education in response to a recent article in the Wall Street Journal from Kenneth Moch, former CEO of Chimerix.

Following recent legislation changes in the US, including the FDA’s streamlined expanded access process and the increasing number of states passing new ‘Right to Try’ laws, Mr Moch is well aware of the pressure that pharma and biotech companies can face in deciding the right response to requests for access to drugs.

Moch is the former CEO of Chimerix, a small US biotech company which, over a matter of days, found itself at the centre of a social media frenzy which made the news around the world in 2014. The family of seven year old Josh Hardy from Virginia approached patient advocacy groups and used social media to pressure Chimerix to provide access to its experimental drug, brincidofovir; Josh’s last hope to fight a life-threatening infection.

Josh received the drug and is now about to celebrate his ninth birthday after unprecedented action from the FDA which enabled Chimerix to initiate a new Phase III clinical trial for which Josh was the first and, for a while, only patient. However, a month after brokering the trial, Moch was replaced as CEO by former CMO M. Michelle Berrey, and left the company.

The media firestorm emphasised the complexity of the global early access space and the ethical questions and dilemmas that pharma and biotech companies now need to address, often as the ultimate decision makers in whether an experimental drug is made available.

Early access: Be prepared!

Although a US-centric incident, the Chimerix case is certainly not a one-off. There is increasing pressure from informed, mobilised patients and their physicians, partly as a result of more transparency of drug-development pipelines online. In the US, requests for expanded access made through the FDA increased by 92% in 2014, a trend that looks set to continue.

The Chimerix experience highlights the need for more education about the options available and the risks and benefits involved in the provision of early access both in the US and much further afield.

Preparedness is key as pressure mounts on corporate management to balance the pressing needs of an individual patient with the longer term potential benefits to a larger population. To have an early access strategy in place that considers and addresses the ethical considerations as well as the logistical needs is important, even if the answer ends up being not to provide access.

In addition, smaller companies developing a single drug or device often struggle most in finding the necessary resources to provide early access. Again, discussion and planning around this area is vital.

Moch has proposed an independent review board to help such companies to consider the associated costs, logistics and anticipated duration of an access program. These companies can also work with specialist providers like Clinigen Global Access Programs (GAP) that provide advice on the viability of a potential program.

In his article, Moch suggests that companies should publically state their policies on early access. Crucially, he believes that a company should also have the right to refuse if “it believes the greater good is served by this decision”. He also recommends that a regulatory framework is put in place to help companies address any unforeseen consequences from granting early access.

Mr Moch’s conclusion – that early access programs are no substitute for clinical trials but that ultimately these programs fulfil an important role for critically ill patients with no other treatment options available to them – is one that I very much agree with. The question is how best to strike that balance.

Having implemented over 120 global access programs for companies ranging from niche biotechs to top 10 pharma, I believe that it is critical to consider early access early, as a core component of the drug development and market access strategy. It is only by understanding the options available, discussing the relative benefits and risks and planning ahead, that corporate management can ensure a successful program. Therefore, educating the industry about ethical access to experimental drugs is essential.

To learn more about early access read the joint BIA and Clinigen GAP blog and post-event report ‘Early access in practice’ from November 2014.